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| Name | Class |
|---|---|
| Accuredit Therapeutics US Limited | INDUSTRY |
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This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental: ART002g1 Injection - Dose Group 1 ( Single IV Infusion) | Experimental | Participants will receive a single dose of ART002g1. |
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| Experimental: ART002g1 Injection - Dose Group 2 (Single IV Infusion) | Experimental | Participants will receive a single dose of ART002g1. |
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| Experimental: ART002g1 Injection - Extended Dose Group ( Single IV Infusion) | Experimental | Participants will receive a single Optimal Biological Dose (OBD) of ART002g1, which is based on the results of the ascending dose escalation. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ART002g1 Injection | Drug | Intravenous (IV) infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | As of Week 48 (W48) post-administration of ART002g1 for Injection |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax | As of Week 2 (W2) post-administration of ART002g1 for Injection | |
| Pharmacodynamic (PD) Assessments: Serum PCSK9 protein | As of Week 48 (W48) post-administration of ART002g1 for Injection |
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Subjects must meet all the following criteria to be eligible for enrollment:
(1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;
(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:
Subjects must not be enrolled if they meet any one or more of the following exclusion criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xueying Ding, MD | Contact | +86-02136126102 | dingxueying@126.com | |
| Rong Jiang, MD | Contact | +86 13795493921 | listening39@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Xueying Ding, MD | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Principal Investigator |
| Rong Jiang, MD | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shanghai General Hospital | Shanghai | China |
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| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax | As of Week 2 (W2) post-administration of ART002g1 for Injection |
| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC | As of Week 2 (W2) post-administration of ART002g1 for Injection |
| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½ | As of Week 2 (W2) post-administration of ART002g1 for Injection |
| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL | As of Week 2 (W2) post-administration of ART002g1 for Injection |
| Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss | As of Week 2 (W2) post-administration of ART002g1 for Injection |
| Pharmacodynamic (PD) Assessments: Serum LDL-C | As of Week 48 (W48) post-administration of ART002g1 for Injection |